The asthma treatment Targacept (NASDAQ:TRGT) is developing actually did not perform as well in clinical trials as initially thought.
Targacept last month released top-line phase 2 results for TC-6987 that showed the asthma compound met both goals in the clinical trial. But the Winston-Salem, North Carolina drug developer has revised its top-line results and said the discovery of a “statistical analysis error” now shows they met just one of the two phase 2 endpoints. Targacept is evaluating its plans for developing TC-6987, which also failed in a separate study as a diabetes treatment. But the revised results of the compound are just the latest bit of bad news for the company, which is refocusing its drug pipeline following the phase 3 clinical trial failure of former lead drug, depression candidate TC-5214.
In the asthma study, TC-6987 was evaluated on its ability to increase an asthma patient’s expiratory volume — the amount of air a patient can blow out after full inspiration. The drug candidate could be used in addition to existing low-dose inhaled corticosteroids or on its own as a separate drug. Targacept had initially reported that the compound met the endpoint of changing expiratory volume from baseline to pre-dosing on day 28 of the four-week study. The company said the compound also met a second endpoint of changing expiratory volume two hours post-dose on day 28 of the study. Reanalysis of the results show that the compound just hit the second target.
Targacept, a spinout from tobacco giant R.J. Reynolds, develops compounds that have an effect on the body’s neuronal nicotinic receptors, or NNRs. Besides TC-6987, the company also has clinical-stage programs in schizophrenia and Alzheimer’s disease. AZD1446, an experimental Alzheimer’s disease treatment, is currently in a mid-stage clinical trial financed and conducted by drug partner AstraZeneca (NYSE:AZN).
Targacept has said that its goal with TC-6987 was to detect a signal of a potential NNR effect in treatments other than central nervous system disorders. The company said TC-6987’s asthma results showed it met that goal. But the company must now determine whether that is enough to continue TC-6987’s development.
“We are in the process of considering potential next steps for this compound and indication,” Targacept President and CEO Donald deBethizy said in a statement. “We regret not having discovered the statistical analysis error initially.”
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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